Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN

Gene therapy by engineered nucleases is a genetic intervention being investigated for curing the hereditary disorders by targeting selected genes with specific nucleotides for establishment, suppression, abolishment of a function or correction of mutation. Here, we review the fast developing technol...

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Veröffentlicht in:Journal of controlled release 2015-05, Vol.205, p.120-127
Hauptverfasser: Ain, Qurrat Ul, Chung, Jee Young, Kim, Yong-Hee
Format: Artikel
Sprache:eng
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Zusammenfassung:Gene therapy by engineered nucleases is a genetic intervention being investigated for curing the hereditary disorders by targeting selected genes with specific nucleotides for establishment, suppression, abolishment of a function or correction of mutation. Here, we review the fast developing technology of targeted genome engineering using site specific programmable nucleases zinc finger nucleases (ZFNs), transcription activator like nucleases (TALENs) and cluster regulatory interspaced short palindromic repeat/CRISPR associated proteins (CRISPR/Cas) based RNA-guided DNA endonucleases (RGENs) and their different characteristics including pros and cons of genome modifications by these nucleases. We have further discussed different types of delivery methods to induce gene editing, novel development in genetic engineering other than nucleases and future prospects. [Display omitted]
ISSN:0168-3659
1873-4995
DOI:10.1016/j.jconrel.2014.12.036